Dear JDCA (Juvenile #Diabetes Care Alliance)… Part 1

Over the past few weeks, I’ve been seeing infographics produced by the Juvenile Diabetes Care Alliance (JDCA) regarding “cure research” and major diabetes organizations.

The infographic is not flattering, and like everything related to statistics, not entirely truthful. (JDCA even admitted in a post on FB that they made some “judgment calls” in their numbers.)

At first, I ignored the repeated posting in Facebook groups of how JDRF and ADA have not done enough to find a cure for us, but after a while, the bashing became nothing but a “I hate these organizations and I’ll never donate to them again. How dare they take our money and do nothing.”

I spoke up and reminded individuals in those groups that there are MANY ways to use the donations raised through walks and rides and other fundraising opportunities, but that ALL of the donations were being used to help people with diabetes, whether it be for research or for support. (And I also reminded them that there was a marked absence in this infographic of where one should be donating…)

My opinion voiced, I went on my merry way, living my life with diabetes, until I got an unsolicited email yesterday from someone claiming to be an employee of JDCA, asking me to promote, on my blog and through social media channels, their petition to the major diabetes non-profits, DEMANDING that JDRF and ADA use 30% of all donations for “practical cure research” through my blog.

Thank you for the invitation, JDCA. I’m going to pass.

Instead, I’m going to take my blog and use it took peek into what your privately funded non-profit, founded by the CEO of Activision Blizzard Entertainment (and a parent of a child diagnosed with Type 1 diabetes at the age of 2) wants us to focus on, rather than telling us what JDRF and ADA isn’t doing.

As I began writing this and hit 2500 words, I realized that this needs to be digested in more than one sitting, so I’m splitting this into 2 blog posts. (Mostly, because no one except masochists read 2500 words of my blog at a time and because I WANT YOU TO READ ALL OF THIS IMPORTANT AND UNBIASED INFORMATION.) Today, I’m covering just the Transplantation “practical cures” and you’ll get the others tomorrow in Part 2.

Juvenile Diabetes Research Alliance

According to a WSJ article, Mr. Kelly’s family foundation, the Brian and Joelle Kelly Family Foundation, has donated $1 million dollars (as of the 2013 article stated) to JDCA. The goal of JDCA is to help donors “become educated about charities that are focused on a “practical cure” for Type 1 diabetes.

Note: Hey, I have no problem with the fact they have spent $1 million dollars to create a “watchdog” organization. They could have spent it on a boat or a house or funding one of their “practical cure” projects. If you’ve got money, you also have the right to spend it as you see fit. I also believe in watchdog organizations, as long as you provide factual, correct, public-facing information.

“Practical Cure”

The Juvenile Diabetes Care Alliance states that they want a “practical cure” by 2025. For their definition, a “practical cure” meets the following four criteria:

Minimal Monitoring (checking BGs less than once per week and a A1C between 5-7%)

Being able to sleep without worrying (about hypoglycemia or hyperglycemia)

Being able to eat whatever one wants without having to evaluate carbohydrate intake

Minimal side effects, understanding that some insignificant side effects are “acceptable”

Sounds reasonable, right? I’m all for that, because that elusive “don’t have to do anything ever again, it’s like magic and you don’t have to think about diabetes for the rest of your life” is a dream.

It then begs the question: What is JDCA’s idea of a practical cure?

JDCA’s Four Ideas of a “practical cure”:

Islet cell transplantation

A device that mimics the pancreas

Glucose-responsive insulin (“smart insulin”)

Modification of the immune system (blocking, balancing, and/or retraining)

Again, sounds reasonable, right? Some of these practical cures are beginning to look like they may not make the 2025 timeframe cut-off, but you never know, right?

Every year, JDCA issues a report that tells potential donors which “practical cures” are more likely to pull ahead. Here are the 2014 “Potential Practical Cure Solutions,” found in the JDCA State of the Cure report. (2015 hasn’t been published yet, but expect it in the fall as in previous years) – and I’m going to show you where the funding is coming from.

JDCA’s 2014 Potential Practical Cure Solutions

These solutions are broken down by type of “practical cure” and gives you the basic info into what it is, where this is in terms of “potential” release into the community and who is funding it. (For my own personal curiosity, I have delved deeper into some than others.) As a reminder, Part 1 is ONLY transplantation. Part 2 is the rest of the “practical cures.”

Transplantation

VC-01

(ViaCyte – a privately held, for profit company)

-San Diego, CA

ViaCyte’s innovative product is based on the differentiation of stem cells into pancreatic beta cell precursors (PEC-01™), with subcutaneous implantation in a retrievable and immune-protective encapsulation medical device (Encaptra® drug delivery system). Once implanted, the precursor cells mature into endocrine cells that secrete insulin and other hormones in a regulated manner to control blood glucose levels. ViaCyte’s goal is a product that can free patients with type 1 and type 2 diabetes from long-term insulin dependence.

Where it is in the pipeline of “practical cure”:

ViaCyte, Inc. announced in July 2014 that it had filed an Investigational New Drug application (IND) with the United States Food and Drug Administration (FDA) seeking to initiate a Phase 1/2 clinical trial in patients with type 1 diabetes, and in August 2014 the IND was accepted, allowing clinical testing to commence. This first-in-human trial will evaluate the safety and efficacy of ViaCyte’s VC-01™ product candidate, a stem cell-derived, encapsulated cell replacement therapy. ( Viacyte’s website)

They are in Phase I/II clinical trials at two locations, with an expected trial end date of August, 2017 with an estimated enrollment of 40 participants. Note: According to Wikipedia, “The percentage of Phase II trials that proceed to Phase III, as of 2008, is 18%.” Estimated commercialization date is unknown, but safe to say more than five years off.

The agreement provides Janssen with a future right to evaluate a transaction related to the VC-01™ combination product that ViaCyte is developing for type 1 diabetes. This right will continue through the initial evaluation of clinical efficacy of VC-01. ViaCyte received $20 million from Janssen and Johnson & Johnson Development Corporation (JJDC). The payment included a rights fee and a note convertible into equity at a later date. JJDC has been a long-standing investor in ViaCyte.

DIABECELL

(Living Cell Technologies – a multinational for-profit in a joint venture with DOL)

– New Zealand, Japan

What it is:

DIABECELL is an insulin-producing cell product derived from pigs for the treatment of type 1 diabetes. These islet cells are self-regulating and efficiently secrete insulin in the patient’s body.

The treatment involves introducing encapsulated pig cells into the patient’s abdomen in a simple laparoscopic procedure. Living Cell Technologies’ unique proprietary encapsulation technology prevents the islet cells from being attacked by the patient’s immune system. This allows the use of cell therapies without the need for co-treatment with drugs that suppress the immune system, which often have negative side effects.

Where it is in the pipeline of “practical cure”:

To date, a total of 46 patients have taken part in clinical trials of DIABECELL. The goals of these trials have been to find the optimal dose required for DIABECELL and to obtain early indication of its effectiveness in controlling type I diabetes. These trials showed that DIABECELL has the potential to significantly reduce the number of unaware hypoglycaemic events that people living with Type I diabetes experience, whilst also reducing their insulin dose and not experiencing a rise in HbA1c.

Phase I/IIa safety study (Russia) – Two out of the eight trial patients discontinued insulin injections for up to 32 weeks. (The assumption being that after 32 weeks, they resumed insulin injections.)

According to Seeking Alpha, an investment media company: “Mostly, the results are more modest involving decreased insulin injections and significant reduction in unaware hypoglycemic events.”

There are currently no clinical trials offered through Clinical Trials.gov (a global trial locator).

Who is funding this “practical cure”?

LCT is an Australian for-profit company with headquarters and operations located in New Zealand. They have a joint venture with Diatranz Otsuka Limited (DOL). In June of 2015, DOL announced:

Diatranz Otsuka Limited (DOL), is to concentrate its research and development activities in supporting the development of DIABECELL® in the United States. As a consequence, research, development and manufacturing of DIABECELL in New Zealand will cease and there will be a reduction of staff at DOL’s headquarters in Auckland.

Otsuka Pharmaceutical Factory, Inc. (OPF), DOL’s 50% shareholder, is full sponsor and funder of the US program, operating under and exclusive license from DOL for US development and commercialisation of DIABECELL. Since securing the license, OPF has made positive progress, establishing a solid partnership framework for the US development.

DOL’s know-how, research to date and clinical experience in pig islet transplantation will be actively combined with OPF’s global drug development expertise and these partnerships to further strengthen and expedite the US development program.

This alignment of DOL’s expertise with the US program is part of DOL’s previously announced commitment to focus on the US development and FDA approval of DIABECELL. Once registered in the US, DOL retains a royalty free right to commercialise the FDA approved product in the rest of the world.

And of greater interest, Seeking Alpha says of DOL:

Otsuka is a formidable Japanese company with an established presence for its products in the US. Most notably its Abilify antipsychotic drug (shared with Bristol-Myers Squibb (NYSE:BMY)) generated $6.5 billion in US sales in 2013 (a top 10 US drug). To show it is no shrinking violet, Otsuka is involved in a fight to keep out generic versions of Abilify by suing the FDA!

None of these companies are non-profits and expressly state that they will sell and commercialize this product. They have not requested funding for cure research from the public.

ßAir Bio-Artifical Pancreas

Beta-O2 Technologies

– Tel Aviv, Israel

What it is:

The ßAir Bio-artificial Pancreas is a macrocapsule that contains islets of Langerhans, cells from pancreatic regions of the body that contain hormone-producing beta-cells. The macrocapsule provides a fully isolated environment for the islets to thrive. The islets in the bio-artificial pancreas replace the function of the endocrine part of the pancreas; i.e., the sensing of the level of glucose in the blood and the regulation of the production of insulin and glucagon as needed.

ßAir has the potential to ‘normalize’ the lives of people living today with type 1 diabetes. How? Of greatest importance, the ßAir bio-artificial pancreas eliminates the need for frequent glucose testing and insulin injections. Immunosuppressive therapy, which has many side effects, is also not required.

Note: as the ßAir device contains ‘living’ cells, the patient must commit to keeping them healthy. Once every 24 hours, the patient needs to refill the air in the device using a replenishing device which includes a dedicated injector. This guarantees that the cells will have the requisite oxygen supply to thrive and perform their insulin and glucagon production role.

Replenishing the device is performed by injecting oxygen into one of two ports implanted under the skin. The replenishing device is very user friendly, requires minimal technical skills for operation, and has very few possibilities for incorrect operation. The replenishing procedure takes just about two minutes. An alarm will trigger if something has gone wrong. (Beta O2 Website)

My Conclusion from Part 1:

For all the talk about JDRF not helping to fund a “practical cure,” I’m here to show you that JDRF is helping. Perhaps not to the extent that JDCA deems acceptable.

Who is JDCA to determine how much is acceptable?

Their “survey” of 1,000 donors in 2014, conducted by a third party (but where did they get their donor responses? Ask yourself that as they didn’t publicly state it in their report) claims that 88% of donors want their donations to go to “cure research.” The questions were targeted to invoke “cure” responses.

And before you jump on me saying that I’m a shill for JDRF (or ADA, who hasn’t been mentioned by me yet)… be very aware of this fact:

I donate to one of the “practical cures” that JDCA touts.

I’ve asked others for their help in funding this “practical cure.” I also donate to other diabetes non-profits, some big and some small, because I know (from my own thirty-two years of T1 diabetes) that while

I would sell everything I own to have a cure, crawl across a minefield strewn with broken glass to not have this horrible disease haunt me…

in the meantime, we need support and research for the complications from this disease.

Every time a parent complains about a school issue in the US, every time a child gets a T1 peer or mentor, every time a teenager goes to a summer camp where diabetes is the norm, every time a family member views their loved one’s BG graph on a device from miles away… those donations matter, too.

Conclusion: DO. YOUR. OWN. RESEARCH.

More information is coming in Part 2, including JDCA’s practical cure solutions: devices (1) and immune system modifications(4).

Don’t be surprised. Or disappointed.

I was told in 1983 there would be a cure in five years. I’ve waited this long. We’ll all be waiting a little longer.

* I have chosen not to link to JDCA’s website. On purpose. They’re easy to Google if you want to see what I did. I’ve linked to the deep dive stuff.

One player you did not include is known as the BetaLogics Venture which is a collaborative venture between the University of British Columbia and J&J’s Janssen unit which is pursuing the same thing that ViaCyte is pursuing (see their website, or more specifically their page at http://jjiv.goldendog.com/IV.html ). I agree in that I’ve given up on JDCA, which seems to do more criticizing of others than pointing out the success they’ve achieved.

I didn’t include them because JDCA didn’t view them as worthy of being a “practical cure” – I simply focused on those mentioned in their reports. This is yet another reason why it’s important for individuals to do their own research. Thank you so much for the link!

I have a very different opinion of JDRF and the ADA. Perhaps it is because I was raised on both as a non PWD and then when I became one it was natural to self identify. Now disagreeing is not the same as disliking. I love your blog for style, substance and passion. The thing is, there are many horses on the range. Someone will get us home. It may be the horse I like or the horse you like. I am rooting for all of them to get on down the road. My grandchildren need them all to work the best they can, to get us across the line. I am counting on it for them.